Page 309 - Demo

Summary / samenvatting30710increasing evidence for differential treatment response and tolerability. For some practitioners, starting treatment with methylphenidate, with blinded crossover periods, the use of placebo and filling out questionnaires is already part of standard care, and debate is ongoing to what extent an N-of-1 study represents medical research or is part of evidence-based care. By comparison, CBD [Epidyolex®] is currently only approved for refractory epilepsy associated with TSC, Lennox-Gastaut syndrome, and Dravet syndrome. Due to recent market approval, evidence is needed for the effectiveness and adverse effects when used to treat behavioral manifestations. If effective, it is yet unclear whether the medication will be approved and reimbursed for this indication in other individuals with RGNDs and ID. Part II: Measuring what mattersAfter identification of treatment targets and drug development, a trial requires appropriately chosen outcome measures to avoid confusion when trial results do not demonstrate benefits, raising the question whether it is due to the inefficacy of the intervention or the chosen outcome measures. For RGNDs and ID, choosing and selecting reliable and valid outcome measurement instruments for trials can be challenging due to the heterogeneity of the populations and cognitive impairments of the individuals. In Chapter 5, an overview of selected outcomes and outcome measurement instruments used in clinical trials in RGNDs, neurometabolic disorders, and ID is provided. Due to the abundancy of instruments and differently used terminology to indicate outcomes, we rather address the overgrowth of outcome measurement instruments to measure the great amount of inconsistently reported outcomes, resulting in a call to action to turn complexity into structure. To illustrate, from 317 clinical trials from the last ten years, we found that 459 different outcome measurement instruments were used of which 290 instruments used in only one clinical trial. Moreover, 438 different outcomes were reported whereas several probably aimed to identify a similar construct. This is problematic for consistency and generalization as well as investigation of measurement properties. Clinical researchers need guidance in choosing appropriate outcome measures Annelieke Muller sHL.indd 307 16-11-2023 09:26